IP Library Granted Patent US 9,439,968
Granted Patent B2
US 9,439,968 · App. 14/987,717 · Granted Sep 13, 2016

Amine-containing lipidoids and uses thereof

Inventors: Daniel Griffith Anderson (Framingham, MA); Kathryn Ann Whitehead (Pittsburgh, PA); Joseph R. Dorkin (Somerville, MA); Arturo Jose Vegas (Belmont, MA); Yunlong Zhang (Cambridge, MA); Robert S. Langer (Newton, MA)
Assignee: Massachusetts Institute of Technology
A61K47/22C07D241/04C07D295/13C12N15/1137C12N2310/14C12N2320/32
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Quick Facts
Patent No.
US 9,439,968
App. No.
14/987,717
Granted
Sep 13, 2016
Kind
B2
Abstract

Provided herein are lipidoids that may be prepared from the conjugate addition of alkylamines to acrylates. In some embodiments, provided lipidoids are biodegradable and may be used in a variety of drug delivery systems. Given the amino moiety of the lipidoids, they are well-suited for the delivery of polynucleotides, in addition to other agents. Nanoparticles containing the inventive lipidoids and polynucleotides have been prepared and have been shown to be effective in delivering siRNA.

Claims (42)

1. A compound of Formula (III):

or a salt thereof, wherein:

each L is, independently, branched or unbranched C 1-6 alkylene, wherein L is optionally substituted with one or more fluorine radicals;

each R is, independently, hydrogen or —CH 2 CH 2 C(═O)OR B ;

each R B is, independently, C 10-14 alkyl, wherein R B is optionally substituted with one or more fluorine radicals;

each R 1 is, independently, fluoro or C 1-6 alkyl optionally substituted with one or more fluorine radicals;

j is 0, 1, 2, 3, or 4; and

p is 1 or 2;

provided that at least three R groups are —CH 2 CH 2 C(═O)OR B .

2. The compound of claim 1 , wherein L is unbranched C 1-6 alkylene.

3. The compound of claim 1 , wherein L is methylene, ethylene, or propylene.

4. The compound of claim 1 , wherein the compound is of the Formula (III-a):

or a salt thereof, wherein each w is, independently, 1, 2, or 3.

5. The compound of claim 1 , wherein p is 1.

6. The compound of claim 1 , wherein p is 2.

7. The compound of claim 4 , wherein the compound is of the formula:

or a salt thereof.

8. The compound of claim 4 , wherein w is 1.

9. The compound of claim 4 , wherein w is 2.

10. The compound of claim 4 , wherein w is 3.

11. The compound of claim 1 , wherein the compound is of the formula:

or a salt thereof.

12. The compound of claim 1 , wherein all R groups are —CH 2 CH 2 C(═O)OR B .

13. The compound of claim 1 , wherein all R B groups are the same.

14. The compound of claim 1 , wherein R B is C 10 alkyl, C 11 alkyl, C 12 alkyl, C 13 alkyl, or C 14 alkyl.

15. The compound of claim 1 , wherein the compound is selected from the group consisting of:

and salts thereof.

16. A nanoparticle comprising a compound of claim 1 and one or more agents to be delivered.

17. The nanoparticle of claim 16 , wherein at least one of the one or more agents is a polynucleotide, drug, protein, peptide, small molecule, or gas.

18. A composition comprising one or more compounds of claim 1 , and an excipient.

19. The composition of claim 18 , wherein the composition further comprises an agent.

20. A method of administering an agent, the method comprising:

administering to a subject in need thereof a therapeutically effective amount of a composition comprising a compound of claim 1 and an agent to be delivered.

21. The nanoparticle of claim 16 , wherein at least one of the one or more agents is DNA or RNA.

22. The composition of claim 19 , wherein the agent is a small molecule or drug.

23. The composition of claim 19 , wherein the agent is a protein or peptide.

24. The composition of claim 19 , wherein the agent is a polynucleotide.

25. The composition of claim 19 , wherein the agent is DNA.

26. The composition of claim 19 , wherein the agent is RNA.

27. The composition of claim 19 , wherein the agent is siRNA, shRNA, mRNA, dsRNA, miRNA, or antisense RNA.

28. The method of claim 20 , wherein the agent is a small molecule, drug, protein, peptide, or polynucleotide.

29. The method of claim 20 , wherein the agent is DNA or RNA.

Assignments (2)
CONFIRMATORY LICENSE Recorded Jun 7, 2018
From: MASSACHUSETTS INSTITUTE OF TECHNOLOGY
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 046317/0282 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jan 29, 2016
From: ANDERSON, DANIEL GRIFFITH; DORKIN, JOSEPH R.; VEGAS, ARTURO JOSE; ZHANG, YUNLONG; LANGER, ROBERT S.; WHITEHEAD, KATHRYN ANN
To: MASSACHUSETTS INSTITUTE OF TECHNOLOGY
Reel/Frame 037617/0763 →
Continuity (4)
Division 14089603 · Nov 25, 2013
Continuation 13966136 · Aug 13, 2013
Provisional Application 61682468 · Aug 13, 2012
Related Publication 20160114042A1 · Apr 28, 2016